US-based biopharmaceutical company BridgeBio Pharma Inc (Nasdaq:BBIO) announced on Wednesday that its affiliate QED Therapeutics has granted Japanese pharmaceutical company Kyowa Kirin Co Ltd (TSE:4151) an exclusive licence to develop and commercialise infigratinib for achondroplasia, hypochondroplasia and other skeletal dysplasias in Japan.

In exchange, BridgeBio will receive a USD100m upfront payment and royalties up to the high-twenties percent on sales of infigratinib in Japan, with additional milestone-based payments. Infigratinib, a once daily oral treatment for achondroplasia, has shown promising efficacy in PROPEL 2, a Phase 2 study in children with achondroplasia.

PROPEL 3, a Phase 3 study of infigratinib in achondroplasia, is underway globally outside of Japan, with the first child dosed in December 2023. This trial builds on the success of PROPEL 2, which demonstrated a significant increase in annualised height velocity. Kyowa Kirin aims to start discussions with the Pharmaceuticals and Medical Devices Agency (PMDA) of Japan in 2024 with the aim of initiating a Japanese registrational trial in 2025.

Achondroplasia affects approximately 55,000 people in the United States and European Union, and 6,000 in Japan. It significantly impacts health and quality of life, leading to various medical complications. The condition is caused by an activating mutation in FGFR3.